[go: up one dir, main page]

WO2014153052A3 - Composition à base d'arnm du gène cftr et procédés et utilisations associés - Google Patents

Composition à base d'arnm du gène cftr et procédés et utilisations associés Download PDF

Info

Publication number
WO2014153052A3
WO2014153052A3 PCT/US2014/028849 US2014028849W WO2014153052A3 WO 2014153052 A3 WO2014153052 A3 WO 2014153052A3 US 2014028849 W US2014028849 W US 2014028849W WO 2014153052 A3 WO2014153052 A3 WO 2014153052A3
Authority
WO
WIPO (PCT)
Prior art keywords
related methods
cftr mrna
mrna compositions
cftr
compositions
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2014/028849
Other languages
English (en)
Other versions
WO2014153052A9 (fr
WO2014153052A2 (fr
Inventor
Michael Heartlein
Braydon Charles Guild
Frank Derosa
Carsten Rudolph
Christian Plank
Lianne SMITH
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ethris GmbH
Shire Human Genetics Therapies Inc
Original Assignee
Ethris GmbH
Shire Human Genetics Therapies Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=50513499&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=WO2014153052(A3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority to SI201430888T priority Critical patent/SI2968586T1/sl
Priority to CN201480015043.5A priority patent/CN105142676B/zh
Priority to NZ711657A priority patent/NZ711657B2/en
Priority to JP2016502924A priority patent/JP6316930B2/ja
Priority to MX2015012333A priority patent/MX365409B/es
Priority to SM20180546T priority patent/SMT201800546T1/it
Priority to CN202210265261.0A priority patent/CN115154620A/zh
Priority to KR1020217031812A priority patent/KR20210122917A/ko
Priority to IL305374A priority patent/IL305374A/en
Priority to PL14718284T priority patent/PL2968586T3/pl
Priority to EP14718284.4A priority patent/EP2968586B1/fr
Priority to KR1020217012925A priority patent/KR102311614B1/ko
Priority to KR1020157023977A priority patent/KR102248744B1/ko
Priority to IL290953A priority patent/IL290953B2/en
Priority to RS20181158A priority patent/RS57739B1/sr
Priority to DK14718284.4T priority patent/DK2968586T3/en
Priority to ES14718284.4T priority patent/ES2689523T3/es
Priority to EA201591477A priority patent/EA037922B1/ru
Priority to SG11201507391VA priority patent/SG11201507391VA/en
Priority to AU2014236305A priority patent/AU2014236305B2/en
Priority to EP18185286.4A priority patent/EP3446712A1/fr
Application filed by Ethris GmbH, Shire Human Genetics Therapies Inc filed Critical Ethris GmbH
Priority to HK16108273.3A priority patent/HK1220137B/en
Priority to LTEP14718284.4T priority patent/LT2968586T/lt
Priority to UAA201508754A priority patent/UA117008C2/uk
Priority to HRP20181580TT priority patent/HRP20181580T1/hr
Priority to CA2904151A priority patent/CA2904151C/fr
Priority to BR112015022868-2A priority patent/BR112015022868B1/pt
Priority to MX2019006294A priority patent/MX393573B/es
Priority to HK16105865.3A priority patent/HK1218068A1/zh
Priority to US14/307,322 priority patent/US9181321B2/en
Publication of WO2014153052A2 publication Critical patent/WO2014153052A2/fr
Publication of WO2014153052A9 publication Critical patent/WO2014153052A9/fr
Priority to IL240982A priority patent/IL240982B/en
Anticipated expiration legal-status Critical
Publication of WO2014153052A3 publication Critical patent/WO2014153052A3/fr
Priority to US14/876,071 priority patent/US9713626B2/en
Priority to US15/625,648 priority patent/US10420791B2/en
Priority to CY181101097T priority patent/CY1120790T1/el
Priority to IL262984A priority patent/IL262984B/en
Priority to AU2019202582A priority patent/AU2019202582B2/en
Priority to US16/540,960 priority patent/US11510937B2/en
Priority to IL282453A priority patent/IL282453B/en
Priority to AU2021215095A priority patent/AU2021215095B2/en
Priority to US18/049,210 priority patent/US20230302039A1/en
Ceased legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • A61K47/6931—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer
    • A61K47/6935—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer the polymer being obtained otherwise than by reactions involving carbon to carbon unsaturated bonds, e.g. polyesters, polyamides or polyglycerol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03049—Channel-conductance-controlling ATPase (3.6.3.49)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Nanotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Pulmonology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Dispersion Chemistry (AREA)
  • Microbiology (AREA)
  • Otolaryngology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cell Biology (AREA)
  • Optics & Photonics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente invention concerne des matériaux, des compositions, des procédés de production et des procédés d'administration d'ARNm du gène CFTR en vue de l'induction de l'expression de la protéine CFTR, y compris dans les poumons d'un mammifère. La présente invention se révèle particulièrement utile dans le cadre du traitement de la mucoviscidose.
PCT/US2014/028849 2013-03-14 2014-03-14 Composition à base d'arnm du gène cftr et procédés et utilisations associés Ceased WO2014153052A2 (fr)

Priority Applications (40)

Application Number Priority Date Filing Date Title
UAA201508754A UA117008C2 (uk) 2013-03-14 2014-03-14 IN VITRO ТРАНСКРИБОВАНА мРНК ТА КОМПОЗИЦІЯ, ЩО ЇЇ МІСТИТЬ, ДЛЯ ЗАСТОСУВАННЯ В ЛІКУВАННІ МУКОВІСЦИДОЗУ В ССАВЦЯ
HK16108273.3A HK1220137B (en) 2013-03-14 2014-03-14 Cftr mrna compositions and related methods and uses
NZ711657A NZ711657B2 (en) 2013-03-14 2014-03-14 Cftr mrna compositions and related methods and uses
JP2016502924A JP6316930B2 (ja) 2013-03-14 2014-03-14 ＣＦＴＲｍＲＮＡ組成物ならびに関連する方法及び使用
MX2015012333A MX365409B (es) 2013-03-14 2014-03-14 Composiciones de ácido ribonucleico mensajero del regulador transmembrana de fibrosis quística y métodos y usos relacionados.
SM20180546T SMT201800546T1 (it) 2013-03-14 2014-03-14 Composizioni di mrna di cftr e metodi e usi correlati
CN202210265261.0A CN115154620A (zh) 2013-03-14 2014-03-14 Cftr mrna组合物以及相关方法和用途
KR1020217031812A KR20210122917A (ko) 2013-03-14 2014-03-14 Cftr mrna 조성물 및 관련 방법 및 사용
IL305374A IL305374A (en) 2013-03-14 2014-03-14 Cftr mrna compositions and related methods and uses
PL14718284T PL2968586T3 (pl) 2013-03-14 2014-03-14 Kompozycje mrna cftr i związne z nimi sposoby i zastosowania
EP14718284.4A EP2968586B1 (fr) 2013-03-14 2014-03-14 Composition à base d'arnm du gène cftr et procédés et utilisations associés
KR1020217012925A KR102311614B1 (ko) 2013-03-14 2014-03-14 Cftr mrna 조성물 및 관련 방법 및 사용
KR1020157023977A KR102248744B1 (ko) 2013-03-14 2014-03-14 Cftr mrna 조성물 및 관련 방법 및 사용
IL290953A IL290953B2 (en) 2013-03-14 2014-03-14 CFTR mRNA Assemblies and Related Methods and Uses
RS20181158A RS57739B1 (sr) 2013-03-14 2014-03-14 Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
CN201480015043.5A CN105142676B (zh) 2013-03-14 2014-03-14 Cftr mrna组合物以及相关方法和用途
ES14718284.4T ES2689523T3 (es) 2013-03-14 2014-03-14 Composiciones a base de ARNM del gen CFTR y métodos y usos relacionados
EA201591477A EA037922B1 (ru) 2013-03-14 2014-03-14 мРНК CFTR И КОМПОЗИЦИИ ДЛЯ ЛЕЧЕНИЯ МУКОВИСЦИДОЗА У МЛЕКОПИТАЮЩЕГО
SG11201507391VA SG11201507391VA (en) 2013-03-14 2014-03-14 Cftr mrna compositions and related methods and uses
AU2014236305A AU2014236305B2 (en) 2013-03-14 2014-03-14 CFTR mRNA compositions and related methods and uses
EP18185286.4A EP3446712A1 (fr) 2013-03-14 2014-03-14 Compositions d'arnm cftr et procédés et utilisations associés
SI201430888T SI2968586T1 (sl) 2013-03-14 2014-03-14 Sestavki CFTR MRNA in z njimi povezani postopki in uporabe
LTEP14718284.4T LT2968586T (lt) 2013-03-14 2014-03-14 Cft mrnr kompozicijos bei su jomis susiję būdai ir panaudojimai
DK14718284.4T DK2968586T3 (en) 2013-03-14 2014-03-14 CFTR MRNA COMPOSITIONS AND RELATED PROCEDURES AND APPLICATIONS
HK16105865.3A HK1218068A1 (zh) 2013-03-14 2014-03-14 Cftr mrna组合物以及相关方法和用途
HRP20181580TT HRP20181580T1 (hr) 2013-03-14 2014-03-14 Pripravci cftr irnk i povezani postupci i upotrebe
CA2904151A CA2904151C (fr) 2013-03-14 2014-03-14 Composition a base d'arnm du gene cftr et procedes et utilisations associes
BR112015022868-2A BR112015022868B1 (pt) 2013-03-14 2014-03-14 Composições de mrna de cftr e usos e métodos relacionados
MX2019006294A MX393573B (es) 2013-03-14 2014-03-14 Composiciones de acido ribonucleico mensajero del regulador transmembrana de fibrosis quistica y metodos y usos relacionados
US14/307,322 US9181321B2 (en) 2013-03-14 2014-06-17 CFTR mRNA compositions and related methods and uses
IL240982A IL240982B (en) 2013-03-14 2015-09-01 mrna cftr compositions related methods and uses
US14/876,071 US9713626B2 (en) 2013-03-14 2015-10-06 CFTR mRNA compositions and related methods and uses
US15/625,648 US10420791B2 (en) 2013-03-14 2017-06-16 CFTR MRNA compositions and related methods and uses
CY181101097T CY1120790T1 (el) 2013-03-14 2018-10-24 Συνθεσεις mrna toy cftr και σχετιζομενες μεθοδοι και χρησεις
IL262984A IL262984B (en) 2013-03-14 2018-11-13 mrna cftr compositions and related methods and uses
AU2019202582A AU2019202582B2 (en) 2013-03-14 2019-04-15 Cftr mrna compositions and related methods and uses
US16/540,960 US11510937B2 (en) 2013-03-14 2019-08-14 CFTR MRNA compositions and related methods and uses
IL282453A IL282453B (en) 2013-03-14 2021-04-20 mrna cftr compositions and related methods and uses
AU2021215095A AU2021215095B2 (en) 2013-03-14 2021-08-09 Cftr mrna compositions and related methods and uses
US18/049,210 US20230302039A1 (en) 2013-03-14 2022-10-24 Cftr mrna compositions and related methods and uses

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361783663P 2013-03-14 2013-03-14
US61/783,663 2013-03-14

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US14/307,322 Continuation US9181321B2 (en) 2013-03-14 2014-06-17 CFTR mRNA compositions and related methods and uses

Publications (3)

Publication Number Publication Date
WO2014153052A2 WO2014153052A2 (fr) 2014-09-25
WO2014153052A9 WO2014153052A9 (fr) 2015-08-06
WO2014153052A3 true WO2014153052A3 (fr) 2015-09-24

Family

ID=50513499

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2014/028849 Ceased WO2014153052A2 (fr) 2013-03-14 2014-03-14 Composition à base d'arnm du gène cftr et procédés et utilisations associés

Country Status (29)

Country Link
US (5) US9181321B2 (fr)
EP (2) EP2968586B1 (fr)
JP (5) JP6316930B2 (fr)
KR (3) KR102248744B1 (fr)
CN (2) CN115154620A (fr)
AU (3) AU2014236305B2 (fr)
BR (1) BR112015022868B1 (fr)
CA (1) CA2904151C (fr)
CL (1) CL2015002675A1 (fr)
CY (1) CY1120790T1 (fr)
DK (1) DK2968586T3 (fr)
EA (2) EA037922B1 (fr)
ES (1) ES2689523T3 (fr)
HK (1) HK1218068A1 (fr)
HR (1) HRP20181580T1 (fr)
HU (1) HUE042640T2 (fr)
IL (5) IL305374A (fr)
LT (1) LT2968586T (fr)
MX (2) MX393573B (fr)
NZ (1) NZ751462A (fr)
PE (1) PE20151773A1 (fr)
PL (1) PL2968586T3 (fr)
PT (1) PT2968586T (fr)
RS (1) RS57739B1 (fr)
SG (2) SG10201710253QA (fr)
SI (1) SI2968586T1 (fr)
SM (1) SMT201800546T1 (fr)
UA (1) UA117008C2 (fr)
WO (1) WO2014153052A2 (fr)

Families Citing this family (65)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3403647A1 (fr) 2009-12-01 2018-11-21 Translate Bio, Inc. Administration d'arnm pour l'augmentation des protéines et des enzymes dans des maladies génétiques humaines
EP2338520A1 (fr) 2009-12-21 2011-06-29 Ludwig Maximilians Universität Conjugué doté d'un liguant de recherche de cible et son utilisation
US8853377B2 (en) * 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US20140206753A1 (en) 2011-06-08 2014-07-24 Shire Human Genetic Therapies, Inc. Lipid nanoparticle compositions and methods for mrna delivery
CA3198966A1 (fr) 2011-06-08 2012-12-13 Translate Bio, Inc. Lipides clivables
EP2859102A4 (fr) 2012-06-08 2016-05-11 Shire Human Genetic Therapies Polynucléotides résistant à la nucléase et leurs utilisations
CA2904151C (fr) 2013-03-14 2023-09-12 Shire Human Genetic Therapies, Inc. Composition a base d'arnm du gene cftr et procedes et utilisations associes
ES2981185T3 (es) 2013-03-14 2024-10-07 Translate Bio Inc Métodos para la purificación de ARN mensajero
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
US20150110857A1 (en) 2013-10-22 2015-04-23 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
WO2015061491A1 (fr) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Thérapie à l'arnm pour la phénylcétonurie
MX373952B (es) 2014-04-25 2020-07-13 Shire Human Genetic Therapies Métodos de purificación de arn mensajero.
CA2979695C (fr) 2015-03-19 2025-02-06 Translate Bio Inc Therapie d'arnm pour maladie de pompe
CN107849565A (zh) 2015-06-30 2018-03-27 埃泽瑞斯公司 Atp结合盒家族的编码多核糖核苷酸及其制剂
WO2017127750A1 (fr) 2016-01-22 2017-07-27 Modernatx, Inc. Acides ribonucléiques messagers pour la production de polypeptides de liaison intracellulaires et leurs procédés d'utilisation
EP3377637B1 (fr) * 2016-04-08 2020-03-18 Krystal Biotech, LLC Compositions pour utilisation dans un procédé destiné au traitement des plaies, des troubles, et des maladies de la peau
JP7150608B6 (ja) * 2016-04-08 2022-11-11 トランスレイト バイオ， インコーポレイテッド 多量体コード核酸及びその使用
US20190167811A1 (en) 2016-04-13 2019-06-06 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
MA45053A (fr) * 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique
KR102469450B1 (ko) 2016-05-18 2022-11-22 모더나티엑스, 인크. 인터류킨-12 (il12)를 코딩하는 폴리뉴클레오티드 및 그의 용도
WO2017205739A1 (fr) 2016-05-26 2017-11-30 University Of Iowa Research Foundation Configurations cis et trans pour la résolution terminale du bocavirus 1 humain
CA3043033A1 (fr) * 2016-11-10 2018-05-17 Translate Bio, Inc. Formulation de nanoparticules lipidiques a base de glace amelioree pour l'administration de l'arnm
JP6730525B2 (ja) 2016-11-21 2020-07-29 ナノストリング テクノロジーズ，インコーポレイティド 化学組成物とそれを利用する方法
SG11201906744VA (en) * 2017-01-27 2019-08-27 Methodist Hospital Core/shell structure platform for immunotherapy
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
ES2925083T3 (es) 2017-02-27 2022-10-13 Translate Bio Inc Métodos de purificación de ARN mensajero
HUE059025T2 (hu) 2017-02-27 2022-10-28 Translate Bio Inc Módszerek a hírvívõ RNS tisztítására
EP3589308A1 (fr) * 2017-02-28 2020-01-08 Sanofi Arn thérapeutique
AU2018246292A1 (en) * 2017-03-31 2019-10-10 Accanis Biotech F&E Gmbh & Co Kg Prevention and treatment of non-melanoma skin cancer (NMSC)
EP3398963B1 (fr) * 2017-05-04 2021-09-29 Eberhard Karls Universität Tübingen Medizinische Fakultät Arnm chimiquement modifié pour une utilisation dans le traitement d'une maladie associée au gène cftr
MA49138A (fr) * 2017-05-16 2020-03-25 Translate Bio Inc Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
US10034951B1 (en) 2017-06-21 2018-07-31 New England Biolabs, Inc. Use of thermostable RNA polymerases to produce RNAs having reduced immunogenicity
EP3437650A1 (fr) 2017-07-31 2019-02-06 Accanis Biotech F&E GmbH & Co KG Traitement de conditions d'hypotrophie cutanées locales
EP3679148A4 (fr) * 2017-09-08 2021-06-09 Generation Bio Co. Formulations de nanoparticules lipidiques de vecteurs d'adn exempts de capside non viraux
CA3094345A1 (fr) 2018-04-12 2019-10-17 Krystal Biotech, Inc. Compositions et procedes pour le traitement d'une ichtyose congenitale autosomique recessive
CN112041434A (zh) 2018-04-27 2020-12-04 克里斯托生物技术股份有限公司 用于美容性应用的编码一种或多种美容蛋白的重组核酸
EP3794146B1 (fr) 2018-05-14 2025-12-10 Bruker Spatial Biology, Inc. Méthode d'identifcation d'une séquence nucléotide predeterminée
CN120514877A (zh) * 2018-07-23 2025-08-22 川斯勒佰尔公司 信使rna的干粉制剂
WO2020041793A1 (fr) 2018-08-24 2020-02-27 Translate Bio, Inc. Procédés de purification d'arn messager
GB2592505B (en) * 2018-09-04 2023-05-03 Univ Texas Compositions and methods for organ specific delivery of nucleic acids
US20230145188A1 (en) * 2018-09-14 2023-05-11 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
JP7562515B2 (ja) 2018-09-24 2024-10-07 クリスタル バイオテック インコーポレイテッド ネザートン症候群の治療のための組成物及び方法
US11072808B2 (en) 2018-10-04 2021-07-27 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed RNA
WO2020072914A1 (fr) 2018-10-04 2020-04-09 New England Biolabs, Inc. Procédés et compositions pour augmenter l'efficacité de coiffage d'un arn transcrit
EP3877538A1 (fr) 2018-11-08 2021-09-15 Translate Bio, Inc. Procédés et compositions pour purification d'arn messager
WO2020097379A2 (fr) * 2018-11-09 2020-05-14 Translate Bio, Inc. Composés polyéthylène glycol à caractère lipidique
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
CN113454105A (zh) * 2019-02-08 2021-09-28 克里斯托生物技术股份有限公司 用于递送cftr多肽的组合物和方法
PT3924487T (pt) 2019-02-11 2024-05-31 Ethris Gmbh Purificação de arnm por filtração de fluxo tangencial
SG11202111353QA (en) * 2019-04-15 2021-11-29 Univ Iowa Res Found Methods and compositions for transgene expression
WO2020214668A1 (fr) * 2019-04-15 2020-10-22 University Of Iowa Research Foundation Compositions et méthodes de traitement de la fibrose kystique
WO2021055609A1 (fr) * 2019-09-20 2021-03-25 Translate Bio, Inc. Arnm codant pour cftr modifiée
WO2021222222A1 (fr) * 2020-04-27 2021-11-04 Mccray Jr Paul B Compositions et procédés pour le traitement de la fibrose kystique
JP2023524071A (ja) * 2020-05-01 2023-06-08 アークトゥラス・セラピューティクス・インコーポレイテッド 嚢胞性線維症を治療するための核酸及び方法
JP7391460B2 (ja) 2020-09-16 2023-12-05 エルジー・ケム・リミテッド 化合物、これを含む抗菌消臭組成物、およびその製造方法
US20220218622A1 (en) 2020-10-14 2022-07-14 George Mason Research Foundation, Inc. Ionizable lipids and methods of manufacture and use thereof
AU2022242828A1 (en) 2021-03-23 2023-10-12 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
JP2024513826A (ja) 2021-04-02 2024-03-27 クリスタル バイオテック インコーポレイテッド がん治療のためのウイルスベクター
WO2022261185A1 (fr) * 2021-06-09 2022-12-15 Recode Therapeutics, Inc. Compositions de polynucléotides, formulations associées et procédés d'utilisation de celles-ci
CN120693181A (zh) 2022-12-08 2025-09-23 瑞科德治疗公司 脂质纳米颗粒组合物及其用途
EP4434534A1 (fr) 2023-03-22 2024-09-25 ADvantage Therapeutics, Inc. Arnm de klotho
WO2025036956A1 (fr) * 2023-08-14 2025-02-20 Ethris Gmbh Administration d'arnm non systémique
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999014346A2 (fr) * 1997-09-19 1999-03-25 Sequitur, Inc. THERAPIES GENIQUES A BASE D'ARNm SENS
WO2007024708A2 (fr) * 2005-08-23 2007-03-01 The Trustees Of The University Of Pennsylvania Arn contenant des nucleosides modifies, et procedes d'utilisation associes
WO2008045548A2 (fr) * 2006-10-12 2008-04-17 Copernicus Therapeutics Inc. Cftr à optimisation par codon
WO2011012316A2 (fr) * 2009-07-31 2011-02-03 Ludwig-Maximilians-Universität Arn ayant une combinaison de nucléotides non modifiés et modifiés pour l'expression protéique
WO2012170930A1 (fr) * 2011-06-08 2012-12-13 Shire Human Genetic Therapies, Inc Compositions de nanoparticules lipides et procédés pour le transfert d'arnm
WO2013182683A1 (fr) * 2012-06-08 2013-12-12 Ethris Gmbh Administration pulmonaire d'un arn messager
WO2013185069A1 (fr) * 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Administration pulmonaire d'arnm à des cellules cibles autres que pulmonaires

Family Cites Families (432)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2647121A (en) 1951-02-02 1953-07-28 Ruth P Jacoby Diamine-bis-acetamides
US2819718A (en) 1953-07-16 1958-01-14 Isidore H Goldman Drainage tube
US2717909A (en) 1953-09-24 1955-09-13 Monsanto Chemicals Hydroxyethyl-keryl-alkylene-ammonium compounds
US2844629A (en) 1956-04-25 1958-07-22 American Home Prod Fatty acid amides and derivatives thereof
US3096560A (en) 1958-11-21 1963-07-09 William J Liebig Process for synthetic vascular implants
GB1072118A (en) 1962-12-01 1967-06-14 Sandoz Ag Amides of aminopropionic acid
JPS5141663B1 (fr) 1966-03-12 1976-11-11
JPS4822365B1 (fr) 1968-10-25 1973-07-05
NL143127B (nl) 1969-02-04 1974-09-16 Rhone Poulenc Sa Versterkingsorgaan voor een defecte hartklep.
US3614955A (en) 1970-02-09 1971-10-26 Medtronic Inc Standby defibrillator and method of operation
US3614954A (en) 1970-02-09 1971-10-26 Medtronic Inc Electronic standby defibrillator
JPS5024216B1 (fr) 1970-12-29 1975-08-14
JPS5012146Y2 (fr) 1971-07-27 1975-04-15
US3945052A (en) 1972-05-01 1976-03-23 Meadox Medicals, Inc. Synthetic vascular graft and method for manufacturing the same
US3805301A (en) 1972-07-28 1974-04-23 Meadox Medicals Inc Tubular grafts having indicia thereon
JPS49127908A (fr) 1973-04-20 1974-12-07
JPS5624664B2 (fr) 1973-06-28 1981-06-08
US4013507A (en) 1973-09-18 1977-03-22 California Institute Of Technology Ionene polymers for selectively inhibiting the vitro growth of malignant cells
JPS5123537A (ja) 1974-04-26 1976-02-25 Adeka Argus Chemical Co Ltd Kasozaisoseibutsu
GB1527592A (en) 1974-08-05 1978-10-04 Ici Ltd Wound dressing
US3995623A (en) 1974-12-23 1976-12-07 American Hospital Supply Corporation Multipurpose flow-directed catheter
JPS5813576B2 (ja) 1974-12-27 1983-03-14 アデカ ア−ガスカガク カブシキガイシヤ 安定化された合成高分子組成物
DE2520814A1 (de) 1975-05-09 1976-11-18 Bayer Ag Lichtstabilisierung von polyurethanen
US4281669A (en) 1975-05-09 1981-08-04 Macgregor David C Pacemaker electrode with porous system
JPS5210847A (en) 1975-07-16 1977-01-27 Nippon Steel Corp Pinch roll
US4096860A (en) 1975-10-08 1978-06-27 Mclaughlin William F Dual flow encatheter
CA1069652A (fr) 1976-01-09 1980-01-15 Alain F. Carpentier Valvule prosthetique avec anneau deformable
US4134402A (en) 1976-02-11 1979-01-16 Mahurkar Sakharam D Double lumen hemodialysis catheter
US4072146A (en) 1976-09-08 1978-02-07 Howes Randolph M Venous catheter device
US4335723A (en) 1976-11-26 1982-06-22 The Kendall Company Catheter having inflatable retention means
US4099528A (en) 1977-02-17 1978-07-11 Sorenson Research Co., Inc. Double lumen cannula
US4140126A (en) 1977-02-18 1979-02-20 Choudhury M Hasan Method for performing aneurysm repair
US4265745A (en) 1977-05-25 1981-05-05 Teijin Limited Permselective membrane
US4182833A (en) 1977-12-07 1980-01-08 Celanese Polymer Specialties Company Cationic epoxide-amine reaction products
US4180068A (en) 1978-04-13 1979-12-25 Motion Control, Incorporated Bi-directional flow catheter with retractable trocar/valve structure
EP0005035B1 (fr) 1978-04-19 1981-09-23 Imperial Chemical Industries Plc Méthode de préparation d'un produit tubulaire par filage électrostatique
US4284459A (en) 1978-07-03 1981-08-18 The Kendall Company Method for making a molded catheter
US4227533A (en) 1978-11-03 1980-10-14 Bristol-Myers Company Flushable urinary catheter
US4375817A (en) 1979-07-19 1983-03-08 Medtronic, Inc. Implantable cardioverter
DE3010841A1 (de) 1980-03-21 1981-10-08 Ulrich Dr.med. 6936 Haag Uthmann Katheder
US4308085A (en) 1980-07-28 1981-12-29 Jenoptik Jena Gmbh Process for the preparation of high molecular thermoplastic epoxide-amine-polyadducts
US4339369A (en) 1981-04-23 1982-07-13 Celanese Corporation Cationic epoxide-amine reaction products
US4406656A (en) 1981-06-01 1983-09-27 Brack Gillium Hattler Venous catheter having collapsible multi-lumens
US4475972A (en) 1981-10-01 1984-10-09 Ontario Research Foundation Implantable material
US4401472A (en) 1982-02-26 1983-08-30 Martin Marietta Corporation Hydraulic cement mixes and processes for improving hydraulic cement mixes
US4568329A (en) 1982-03-08 1986-02-04 Mahurkar Sakharam D Double lumen catheter
US4546499A (en) 1982-12-13 1985-10-15 Possis Medical, Inc. Method of supplying blood to blood receiving vessels
US4530113A (en) 1983-05-20 1985-07-23 Intervascular, Inc. Vascular grafts with cross-weave patterns
US4550447A (en) 1983-08-03 1985-11-05 Shiley Incorporated Vascular graft prosthesis
US4647416A (en) 1983-08-03 1987-03-03 Shiley Incorporated Method of preparing a vascular graft prosthesis
US5104399A (en) 1986-12-10 1992-04-14 Endovascular Technologies, Inc. Artificial graft and implantation method
US4571241A (en) 1983-12-16 1986-02-18 Christopher T Graham Urinary catheter with collapsible urethral tube
US4710169A (en) 1983-12-16 1987-12-01 Christopher T Graham Urinary catheter with collapsible urethral tube
US4737518A (en) 1984-04-03 1988-04-12 Takeda Chemical Industries, Ltd. Lipid derivatives, their production and use
US4562596A (en) 1984-04-25 1986-01-07 Elliot Kornberg Aortic graft, device and method for performing an intraluminal abdominal aortic aneurysm repair
US4782836A (en) 1984-05-24 1988-11-08 Intermedics, Inc. Rate adaptive cardiac pacemaker responsive to patient activity and temperature
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4662382A (en) 1985-01-16 1987-05-05 Intermedics, Inc. Pacemaker lead with enhanced sensitivity
US4762915A (en) 1985-01-18 1988-08-09 Liposome Technology, Inc. Protein-liposome conjugates
US4860751A (en) 1985-02-04 1989-08-29 Cordis Corporation Activity sensor for pacemaker control
US5223263A (en) 1988-07-07 1993-06-29 Vical, Inc. Liponucleotide-containing liposomes
CA1320724C (fr) 1985-07-19 1993-07-27 Koichi Kanehira Aminoalcools terpeniques et leurs utilisations medicales
US4701162A (en) 1985-09-24 1987-10-20 The Kendall Company Foley catheter assembly
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
DE3616824A1 (de) 1986-05-17 1987-11-19 Schering Ag Verwendung von haertbaren kunstharzmischungen fuer oberflaechenbeschichtungen und druckfarben und verfahren zu ihrer herstellung
DE3780374D1 (de) 1986-07-31 1992-08-20 Irnich Werner Frequenzadaptierender herzschrittmacher.
US4960409A (en) 1986-09-11 1990-10-02 Catalano Marc L Method of using bilumen peripheral venous catheter with adapter
JPH0829776B2 (ja) 1986-10-29 1996-03-27 東燃化学株式会社 合成樹脂製容器及びその製造用金型
US4720517A (en) 1986-11-24 1988-01-19 Ciba-Geigy Corporation Compositions stabilized with N-hydroxyiminodiacetic and dipropionic acids and esters thereof
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
JPS63154788U (fr) 1987-03-31 1988-10-11
DE3728917A1 (de) 1987-08-29 1989-03-09 Roth Hermann J Neue lipide mit unsymmetrisch substituierter disulfidbruecke
US4946683A (en) 1987-11-18 1990-08-07 Vestar, Inc. Multiple step entrapment/loading procedure for preparing lipophilic drug-containing liposomes
US5138067A (en) 1987-12-17 1992-08-11 Shionogi & Co. Ltd. Lipid derivatives
US5047540A (en) 1987-12-17 1991-09-10 Shionogi & Co., Ltd. Lipid derivatives
US4892540A (en) 1988-04-21 1990-01-09 Sorin Biomedica S.P.A. Two-leaflet prosthetic heart valve
US5176661A (en) 1988-09-06 1993-01-05 Advanced Cardiovascular Systems, Inc. Composite vascular catheter
US5024671A (en) 1988-09-19 1991-06-18 Baxter International Inc. Microporous vascular graft
US5200395A (en) 1988-10-18 1993-04-06 Ajinomoto Company, Inc. Pharmaceutical composition of BUF-5 for treating anemia
CA2001401A1 (fr) 1988-10-25 1990-04-25 Claude Piantadosi Derives d'ether ou d'ester lipidique contenant une amine quaternaire et composes therapeutiques
US6214804B1 (en) 1989-03-21 2001-04-10 Vical Incorporated Induction of a protective immune response in a mammal by injecting a DNA sequence
DE69034168T3 (de) 1989-03-21 2013-04-11 Vical, Inc. Expression von exogenen Polynukleotidsequenzen in Wirbeltieren
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5194654A (en) 1989-11-22 1993-03-16 Vical, Inc. Lipid derivatives of phosphonoacids for liposomal incorporation and method of use
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5101824A (en) 1990-04-16 1992-04-07 Siemens-Pacesetter, Inc. Rate-responsive pacemaker with circuitry for processing multiple sensor inputs
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
EP0549590A1 (fr) 1990-07-26 1993-07-07 LANE, Rodney James Endoprothese vasculaire a autodilatation pour anevrismes
US5693338A (en) 1994-09-29 1997-12-02 Emisphere Technologies, Inc. Diketopiperazine-based delivery systems
DE9117152U1 (de) 1990-10-09 1996-07-11 Cook Inc., Bloomington, Ind. Stent
ATE120971T1 (de) 1990-12-19 1995-04-15 Osypka Peter Herzschrittmacherleitung mit einem inneren kanal und mit einem elektrodenkopf.
US5116360A (en) 1990-12-27 1992-05-26 Corvita Corporation Mesh composite graft
US5405363A (en) 1991-03-15 1995-04-11 Angelon Corporation Implantable cardioverter defibrillator having a smaller displacement volume
US5330768A (en) 1991-07-05 1994-07-19 Massachusetts Institute Of Technology Controlled drug delivery using polymer/pluronic blends
US6013638A (en) * 1991-10-02 2000-01-11 The United States Of America As Represented By The Department Of Health And Human Services Adenovirus comprising deletions on the E1A, E1B and E3 regions for transfer of genes to the lung
US5545449A (en) 1991-10-02 1996-08-13 Weyerhaeuser Company Polyether-reinforced fiber-based materials
US5151105A (en) 1991-10-07 1992-09-29 Kwan Gett Clifford Collapsible vessel sleeve implant
JPH0753535B2 (ja) 1992-02-14 1995-06-07 株式会社カワタ 粉粒体の材料供給装置における弁装置
US5284491A (en) 1992-02-27 1994-02-08 Medtronic, Inc. Cardiac pacemaker with hysteresis behavior
US5352461A (en) 1992-03-11 1994-10-04 Pharmaceutical Discovery Corporation Self assembling diketopiperazine drug delivery system
SE9200951D0 (sv) 1992-03-27 1992-03-27 Kabi Pharmacia Ab Pharmaceutical composition containing a defined lipid system
EP0635019B1 (fr) 1992-04-06 1999-05-26 Biosite Diagnostics Inc. Derives opiaces et etiquettes et conjugues de derives opiaces polypeptidiques et proteiques
US6670178B1 (en) 1992-07-10 2003-12-30 Transkaryotic Therapies, Inc. In Vivo production and delivery of insulinotropin for gene therapy
WO1994003598A1 (fr) 1992-08-04 1994-02-17 The Green Cross Corporation Agent antiallergique
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5461223A (en) 1992-10-09 1995-10-24 Eastman Kodak Company Bar code detecting circuitry
US5300022A (en) 1992-11-12 1994-04-05 Martin Klapper Urinary catheter and bladder irrigation system
US5496362A (en) 1992-11-24 1996-03-05 Cardiac Pacemakers, Inc. Implantable conformal coil patch electrode with multiple conductive elements for cardioversion and defibrillation
US5552155A (en) 1992-12-04 1996-09-03 The Liposome Company, Inc. Fusogenic lipsomes and methods for making and using same
US5716395A (en) 1992-12-11 1998-02-10 W.L. Gore & Associates, Inc. Prosthetic vascular graft
KR100334858B1 (ko) 1993-02-19 2006-01-27 니뽄 신야쿠 가부시키가이샤 핵산공중합체를함유하는의약조성물
US5395619A (en) 1993-03-03 1995-03-07 Liposome Technology, Inc. Lipid-polymer conjugates and liposomes
US5697953A (en) 1993-03-13 1997-12-16 Angeion Corporation Implantable cardioverter defibrillator having a smaller displacement volume
US5624976A (en) 1994-03-25 1997-04-29 Dentsply Gmbh Dental filling composition and method
US5314430A (en) 1993-06-24 1994-05-24 Medtronic, Inc. Atrial defibrillator employing transvenous and subcutaneous electrodes and method of use
DE4325848A1 (de) 1993-07-31 1995-02-02 Basf Ag Verfahren zur Herstellung von N-(2-Hydroxyethyl)-piperazin
DE69428057T2 (de) 1993-10-06 2002-04-18 Mitsubishi Jukogyo K.K., Tokio/Tokyo Verfahren zur Abscheidung von Kohlendioxid aus Verbrennungsabgasen
US5609624A (en) 1993-10-08 1997-03-11 Impra, Inc. Reinforced vascular graft and method of making same
SE9303481L (sv) 1993-10-22 1995-04-23 Berol Nobel Ab Hygienkomposition
AU1091095A (en) 1993-11-08 1995-05-29 Harrison M. Lazarus Intraluminal vascular graft and method
JPH09505593A (ja) 1993-11-24 1997-06-03 メガバイオス・コーポレイション ピペラジンの両親媒性誘導体
US5595756A (en) 1993-12-22 1997-01-21 Inex Pharmaceuticals Corporation Liposomal compositions for enhanced retention of bioactive agents
US5464924A (en) 1994-01-07 1995-11-07 The Dow Chemical Company Flexible poly(amino ethers) for barrier packaging
US5844107A (en) 1994-03-23 1998-12-01 Case Western Reserve University Compacted nucleic acids and their delivery to cells
NZ268146A (en) 1994-04-12 1997-10-24 Liposome Co Inc Liposome compositions and medicinal uses
US5776747A (en) 1994-07-20 1998-07-07 Cytotherapeutics, Inc. Method for controlling the distribution of cells within a bioartificial organ using polycthylene oxide-poly (dimethylsiloxane) copolymer
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5641665A (en) 1994-11-28 1997-06-24 Vical Incorporated Plasmids suitable for IL-2 expression
US6071890A (en) 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
US5965434A (en) 1994-12-29 1999-10-12 Wolff; Jon A. Amphipathic PH sensitive compounds and delivery systems for delivering biologically active compounds
US6485726B1 (en) 1995-01-17 2002-11-26 The Brigham And Women's Hospital, Inc. Receptor specific transepithelial transport of therapeutics
US5830430A (en) 1995-02-21 1998-11-03 Imarx Pharmaceutical Corp. Cationic lipids and the use thereof
EP0822835A1 (fr) 1995-04-17 1998-02-11 Imarx Pharmaceutical Corp. Agents de contraste hybrides pour la resonance magnetique nucleaire
US5772694A (en) 1995-05-16 1998-06-30 Medical Carbon Research Institute L.L.C. Prosthetic heart valve with improved blood flow
US5783383A (en) 1995-05-23 1998-07-21 The Board Of Trustees Of The Leland Stanford Junior University Method of detecting cytomegalovirus (CMV)
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5609629A (en) 1995-06-07 1997-03-11 Med Institute, Inc. Coated implantable medical device
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
DE69634084T2 (de) 1995-06-07 2005-12-08 Inex Pharmaceuticals Corp. Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
US5607385A (en) 1995-08-17 1997-03-04 Medtronic, Inc. Device and algorithm for a combined cardiomyostimulator and a cardiac pacer-carioverter-defibrillator
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
FR2740978B1 (fr) 1995-11-10 1998-01-02 Ela Medical Sa Dispositif medical actif du type defibrillateur/cardioverteur implantable
US5874105A (en) 1996-01-31 1999-02-23 Collaborative Laboratories, Inc. Lipid vesicles formed with alkylammonium fatty acid salts
CA2252055C (fr) 1996-04-11 2007-01-16 The University Of British Columbia Liposomes entrainant une fusion
US5935936A (en) 1996-06-03 1999-08-10 Genzyme Corporation Compositions comprising cationic amphiphiles and co-lipids for intracellular delivery of therapeutic molecules
US5913848A (en) 1996-06-06 1999-06-22 Luther Medical Products, Inc. Hard tip over-the-needle catheter and method of manufacturing the same
US5677124A (en) 1996-07-03 1997-10-14 Ambion, Inc. Ribonuclease resistant viral RNA standards
US5736573A (en) 1996-07-31 1998-04-07 Galat; Alexander Lipid and water soluble derivatives of drugs
US7288266B2 (en) 1996-08-19 2007-10-30 United States Of America As Represented By The Secretary, Department Of Health And Human Services Liposome complexes for increased systemic delivery
CA2264140A1 (fr) 1996-08-26 1998-03-05 Transgene S.A. Complexes acides nucleiques/lipides cationiques
ES2305157T3 (es) 1996-09-13 2008-11-01 Lipoxen Technologies Limited Liposomas.
TW520297B (en) 1996-10-11 2003-02-11 Sequus Pharm Inc Fusogenic liposome composition and method
DE69735382T2 (de) 1996-11-04 2006-11-30 Qiagen Gmbh Kationische reagenzien zür transfektion
US6887665B2 (en) 1996-11-14 2005-05-03 Affymetrix, Inc. Methods of array synthesis
US5985930A (en) 1996-11-21 1999-11-16 Pasinetti; Giulio M. Treatment of neurodegenerative conditions with nimesulide
US6204297B1 (en) 1996-11-26 2001-03-20 Rhodia Inc. Nonionic gemini surfactants
JPH10197978A (ja) 1997-01-09 1998-07-31 Mitsubishi Paper Mills Ltd ハロゲン化銀写真感光材料
EP0853123A1 (fr) 1997-01-10 1998-07-15 Roche Diagnostics GmbH Purification d'ADN à l'aide de 'cross-flow-filtration'
FR2760193B1 (fr) 1997-02-28 1999-05-28 Transgene Sa Lipides et complexes de lipides cationiques et de substances actives, notamment pour la transfection de cellules
US5837283A (en) 1997-03-12 1998-11-17 The Regents Of The University Of California Cationic lipid compositions targeting angiogenic endothelial cells
US5945326A (en) 1997-03-20 1999-08-31 New England Biolabs, Inc. Method for cloning and producing the Spel restriction endonuclease
US20030104044A1 (en) 1997-05-14 2003-06-05 Semple Sean C. Compositions for stimulating cytokine secretion and inducing an immune response
DE69841002D1 (de) 1997-05-14 2009-09-03 Univ British Columbia Hochwirksame verkapselung von nukleinsäuren in lipidvesikeln
US6835395B1 (en) 1997-05-14 2004-12-28 The University Of British Columbia Composition containing small multilamellar oligodeoxynucleotide-containing lipid vesicles
JPH115786A (ja) 1997-06-13 1999-01-12 Pola Chem Ind Inc 新規アミノヒドロキシプロピルピペラジン誘導体
US6067471A (en) 1998-08-07 2000-05-23 Cardiac Pacemakers, Inc. Atrial and ventricular implantable cardioverter-defibrillator and lead system
JPH1180142A (ja) 1997-09-05 1999-03-26 Pola Chem Ind Inc ジフェニルアルキル化合物の製造法
US6165763A (en) 1997-10-30 2000-12-26 Smithkline Beecham Corporation Ornithine carbamoyltransferase
US6096075A (en) 1998-01-22 2000-08-01 Medical Carbon Research Institute, Llc Prosthetic heart valve
US6617171B2 (en) 1998-02-27 2003-09-09 The General Hospital Corporation Methods for diagnosing and treating autoimmune disease
US6271209B1 (en) 1998-04-03 2001-08-07 Valentis, Inc. Cationic lipid formulation delivering nucleic acid to peritoneal tumors
US6176877B1 (en) 1998-04-20 2001-01-23 St. Jude Medical, Inc. Two piece prosthetic heart valve
DE19822602A1 (de) 1998-05-20 1999-11-25 Goldschmidt Ag Th Verfahren zur Herstellung von Polyaminosäureestern durch Veresterung von sauren Polyaminosäuren oder Umesterung von Polyaminosäureestern
NO313244B1 (no) 1998-07-08 2002-09-02 Crew Dev Corp Fremgangsmåte for isolering og produksjon av magnesitt eller magnesiumklorid
US6055454A (en) 1998-07-27 2000-04-25 Cardiac Pacemakers, Inc. Cardiac pacemaker with automatic response optimization of a physiologic sensor based on a second sensor
US6312926B1 (en) 1998-08-14 2001-11-06 University Of Medicine & Dentistry Of New Jersey mRNA capping enzymes and uses thereof
CA2340652C (fr) 1998-08-20 2013-09-24 Cook Incorporated Dispositif medical implantable dote d'un revetement et comprenant du paclitaxel
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6468793B1 (en) * 1998-10-23 2002-10-22 Florida State University Research Foundation CFTR genes and proteins for cystic fibrosis gene therapy
US6656498B1 (en) 1998-11-25 2003-12-02 Vanderbilt University Cationic liposomes for gene transfer
US6248725B1 (en) 1999-02-23 2001-06-19 Amgen, Inc. Combinations and methods for promoting in vivo liver cell proliferation and enhancing in vivo liver-directed gene transduction
US6379698B1 (en) 1999-04-06 2002-04-30 Isis Pharmaceuticals, Inc. Fusogenic lipids and vesicles
AU783647B2 (en) 1999-04-20 2005-11-17 University Of British Columbia, The Cationic peg-lipids and methods of use
EP1880736A1 (fr) 1999-04-23 2008-01-23 Alza Corporation Liaison amovible et composition la contenant
US6169923B1 (en) 1999-04-23 2001-01-02 Pacesetter, Inc. Implantable cardioverter-defibrillator with automatic arrhythmia detection criteria adjustment
MXPA01011778A (es) * 1999-05-17 2002-11-04 Edscha Ag Arreglo de cofre o capo frontal.
ATE369384T1 (de) 1999-05-19 2007-08-15 Emd Lexigen Res Ct Corp Expression und export von interferon-alpha proteinen als fc fusionsproteine
US6696424B1 (en) 1999-05-28 2004-02-24 Vical Incorporated Cytofectin dimers and methods of use thereof
US6346382B1 (en) 1999-06-01 2002-02-12 Vanderbilt University Human carbamyl phosphate synthetase I polymorphism and diagnostic methods related thereto
WO2001005375A1 (fr) 1999-07-16 2001-01-25 Purdue Research Foundation Lipides d'ether vinyliques avec des groupes de tetes hydrophiles clivables
BR0012627A (pt) 1999-07-23 2002-04-09 Genentech Inc Processo para purificar o dna de plasmìdeo de células procarióticas mediante o emprego de filtração de fluxo tangencial
US6358278B1 (en) 1999-09-24 2002-03-19 St. Jude Medical, Inc. Heart valve prosthesis with rotatable cuff
US6371983B1 (en) 1999-10-04 2002-04-16 Ernest Lane Bioprosthetic heart valve
US7060291B1 (en) 1999-11-24 2006-06-13 Transave, Inc. Modular targeted liposomal delivery system
CN101041079A (zh) 1999-12-30 2007-09-26 诺瓦提斯公司 用于基因治疗的新的胶体合成载体
CA2400634A1 (fr) 2000-02-17 2001-08-23 Chester Li Modification genetique du poumon utilise comme porte d'entree pour l'administration de genes
US6370434B1 (en) 2000-02-28 2002-04-09 Cardiac Pacemakers, Inc. Cardiac lead and method for lead implantation
US6565960B2 (en) 2000-06-01 2003-05-20 Shriners Hospital Of Children Polymer composite compositions
WO2002000870A2 (fr) 2000-06-26 2002-01-03 Christian Plank Procede de transfection de cellules a l'aide d'un champ magnetique
IL138474A0 (en) 2000-09-14 2001-10-31 Epox Ltd Highly branched water-soluble polyamine oligomers, process for their preparation and applications thereof
US7427394B2 (en) 2000-10-10 2008-09-23 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
USRE43612E1 (en) 2000-10-10 2012-08-28 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
US6998115B2 (en) 2000-10-10 2006-02-14 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
AU2002214854A1 (en) 2000-10-25 2002-05-06 Inex Pharmaceuticals Corporation Lipid formulations for target delivery
GB0028361D0 (en) 2000-11-21 2001-01-03 Glaxo Group Ltd Method of separating extra chromosomal dna from other cellular components
US20020094528A1 (en) 2000-11-29 2002-07-18 Salafsky Joshua S. Method and apparatus using a surface-selective nonlinear optical technique for detection of probe-target interations
JP2002167368A (ja) 2000-12-01 2002-06-11 Nitto Denko Corp アルキル置換デンドリマーおよびその製造法
US20050004058A1 (en) 2000-12-07 2005-01-06 Patrick Benoit Sequences upstream of the carp gene, vectors containing them and uses thereof
DE10109897A1 (de) 2001-02-21 2002-11-07 Novosom Ag Fakultativ kationische Liposomen und Verwendung dieser
US20020192721A1 (en) 2001-03-28 2002-12-19 Engeneos, Inc. Modular molecular clasps and uses thereof
TW588032B (en) 2001-04-23 2004-05-21 Shinetsu Chemical Co New tertiary amine compound having ester structure and method for producing the same
US6585410B1 (en) 2001-05-03 2003-07-01 The United States Of America As Represented By The Administrator Of The National Aeronautics And Space Administration Radiant temperature nulling radiometer
DE50214201D1 (de) 2001-06-05 2010-03-25 Curevac Gmbh Stabilisierte mRNA mit erhöhtem G/C-Gehalt, enkodierend für ein bakterielles Antigen sowie deren Verwendung
EP2390328A1 (fr) 2001-09-28 2011-11-30 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Molécules de micro ARN
EP1446665A2 (fr) 2001-11-09 2004-08-18 Bayer HealthCare AG Marqueurs d'affinite 3 a codage isotopique
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
DE10207178A1 (de) 2002-02-19 2003-09-04 Novosom Ag Komponenten für die Herstellung amphoterer Liposomen
DE10214983A1 (de) 2002-04-04 2004-04-08 TransMIT Gesellschaft für Technologietransfer mbH Vernebelbare Liposomen und ihre Verwendung zur pulmonalen Applikation von Wirkstoffen
US20030215395A1 (en) 2002-05-14 2003-11-20 Lei Yu Controllably degradable polymeric biomolecule or drug carrier and method of synthesizing said carrier
US7601367B2 (en) 2002-05-28 2009-10-13 Mirus Bio Llc Compositions and processes using siRNA, amphipathic compounds and polycations
WO2003102140A2 (fr) * 2002-05-31 2003-12-11 Children's Hospital Medical Center Genes modificateurs cftr et polypeptides exprimes utiles pour traiter la mucoviscidose et techniques et produits permettant de detecter et/ou d'identifier ceux-ci
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
DE10229872A1 (de) 2002-07-03 2004-01-29 Curevac Gmbh Immunstimulation durch chemisch modifizierte RNA
US20040028804A1 (en) 2002-08-07 2004-02-12 Anderson Daniel G. Production of polymeric microarrays
US20050244961A1 (en) 2002-08-22 2005-11-03 Robert Short Cell culture surface
EP1565512B1 (fr) 2002-11-04 2018-07-04 Momentive Performance Materials GmbH Copolymères polyamino-polysiloxane et/ou polyammonium-polysiloxane linéaires i
JP2006515574A (ja) 2002-11-22 2006-06-01 ノボ・ノルデイスク・エー／エス 肥満症の治療に使用される化合物
US7169892B2 (en) 2003-01-10 2007-01-30 Astellas Pharma Inc. Lipid-peptide-polymer conjugates for long blood circulation and tumor specific drug delivery systems
US8054291B2 (en) 2003-01-20 2011-11-08 Asahi Kasei Emd Corporation Pointing device
NZ542665A (en) 2003-03-05 2008-05-30 Senesco Technologies Inc Use of antisense oligonucleotides or siRNA to suppress expression of eIF-5A1
US20040224912A1 (en) 2003-05-07 2004-11-11 Isis Pharmaceuticals Inc. Modulation of PAI-1 mRNA-binding protein expression
US7619017B2 (en) 2003-05-19 2009-11-17 Wacker Chemical Corporation Polymer emulsions resistant to biodeterioration
WO2005007810A2 (fr) 2003-06-16 2005-01-27 Grinstaff Mark W Macromolecules et molecules synthetiques fonctionnelles pour l'administration de genes
EP1675943A4 (fr) 2003-09-15 2007-12-05 Massachusetts Inst Technology Synthese, a l'echelle du nanolitre, de biomateriaux en reseaux et criblage de ceux-ci
EP1664316B1 (fr) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Composes lipidiques modifies avec du polyethyleneglycol et utilisations de ces composes
US20050069590A1 (en) 2003-09-30 2005-03-31 Buehler Gail K. Stable suspensions for medicinal dosages
ES2457022T3 (es) 2003-10-10 2014-04-24 Powderject Vaccines, Inc. Construcciones de ácidos nucleicos
WO2005037226A2 (fr) 2003-10-17 2005-04-28 Georgia Tech Research Corporation Cellules enteroendocrines genetiquement modifiees pour le traitement des troubles metaboliques lies au glucose
WO2005044782A1 (fr) 2003-11-10 2005-05-19 Nippon Kayaku Kabushiki Kaisha Compose de sel de diimonium et utilisation
US7022214B2 (en) 2004-01-21 2006-04-04 Bio-Rad Laboratories, Inc. Carrier ampholytes of high pH range
US7556684B2 (en) 2004-02-26 2009-07-07 Construction Research & Technology Gmbh Amine containing strength improvement admixture
US20060228404A1 (en) 2004-03-04 2006-10-12 Anderson Daniel G Compositions and methods for treatment of hypertrophic tissues
US20090069256A1 (en) 2004-06-04 2009-03-12 Smith Larry R Enhancing protein expression
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
AU2005252273B2 (en) 2004-06-07 2011-04-28 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
US7670595B2 (en) 2004-06-28 2010-03-02 Merck Patent Gmbh Fc-interferon-beta fusion proteins
GB0418172D0 (en) 2004-08-13 2004-09-15 Ic Vec Ltd Vector
DE102004042546A1 (de) 2004-09-02 2006-03-09 Curevac Gmbh Kombinationstherapie zur Immunstimulation
DE102004043342A1 (de) 2004-09-08 2006-03-09 Bayer Materialscience Ag Blockierte Polyurethan-Prepolymere als Klebstoffe
CA2586708A1 (fr) 2004-11-05 2006-05-11 Novosom Ag Ameliorations apportees a des compositions pharmaceutiques comprenant un oligonucleotide comme agent actif
GB0502482D0 (en) 2005-02-07 2005-03-16 Glaxo Group Ltd Novel compounds
WO2007086883A2 (fr) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Compositions à base de nanoparticules lipidiques et méthodes pour l'administration de molécules biologiquement actives
WO2006105043A2 (fr) 2005-03-28 2006-10-05 Dendritic Nanotechnologies, Inc. Dendrimeres et dendrons de type janus
WO2006138380A2 (fr) 2005-06-15 2006-12-28 Massachusetts Institute Of Technology Lipides contenant des amines et utilisations
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
WO2007031091A2 (fr) 2005-09-15 2007-03-22 Santaris Pharma A/S Composes antagonistes d'arn de modulation de l'expression de p21 ras
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
US7238791B1 (en) 2005-12-16 2007-07-03 Roche Diagnostics Operations, Inc. 6-monoacetylmorphine derivatives useful in immunoassay
WO2007073489A2 (fr) 2005-12-22 2007-06-28 Trustees Of Boston University Molécules pour administration de gènes et thérapie génique et méthodes d'utilisation de celles-ci
CN100569877C (zh) 2005-12-30 2009-12-16 财团法人工业技术研究院 含多uv交联反应基的分枝状结构化合物及其应用
CA2649325C (fr) 2006-04-14 2019-01-08 Epicentre Technologies Corporation Kits et procedes pour la generation d'arn coiffe en 5'
US9085778B2 (en) 2006-05-03 2015-07-21 VL27, Inc. Exosome transfer of nucleic acids to cells
MX2008014437A (es) * 2006-05-19 2008-11-27 Scripps Research Inst Tratamiento de desplegamiento de proteinas.
US20070275923A1 (en) 2006-05-25 2007-11-29 Nastech Pharmaceutical Company Inc. CATIONIC PEPTIDES FOR siRNA INTRACELLULAR DELIVERY
US8808681B2 (en) 2006-06-05 2014-08-19 Massachusetts Institute Of Technology Crosslinked, degradable polymers and uses thereof
US20090186805A1 (en) 2006-07-06 2009-07-23 Aaron Thomas Tabor Compositions and Methods for Genetic Modification of Cells Having Cosmetic Function to Enhance Cosmetic Appearance
ES2293834B1 (es) 2006-07-20 2009-02-16 Consejo Superior Investig. Cientificas Compuesto con actividad inhibidora de las interacciones ubc13-uev, composiciones farmaceuticas que lo comprenden y sus aplicaciones terapeuticas.
WO2008011561A2 (fr) 2006-07-21 2008-01-24 Massachusetts Institute Of Technology Poly(béta-amino esters) à extrémité modifiée et leurs utilisations
CA2927045A1 (fr) 2006-10-03 2008-04-10 Muthiah Manoharan Formulations contenant un lipide
DE102006051516A1 (de) 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
CN101611145B (zh) 2006-12-21 2013-08-14 诺维信股份有限公司 用于在细菌细胞中表达基因的修饰型信使rna稳定化序列
DE102007001370A1 (de) 2007-01-09 2008-07-10 Curevac Gmbh RNA-kodierte Antikörper
US8859229B2 (en) 2007-02-02 2014-10-14 Yale University Transient transfection with RNA
WO2008113364A2 (fr) 2007-03-20 2008-09-25 Recepticon Aps Prévention de la néphrotoxicité iii
WO2008121199A2 (fr) * 2007-03-28 2008-10-09 University Of Iowa Research Foundation Modèles d'animaux transgéniques pour une maladie
JP5186126B2 (ja) 2007-03-29 2013-04-17 公益財団法人地球環境産業技術研究機構 新規トリアジン誘導体ならびにその製法およびそのガス分離膜としての用途
CN101820875B (zh) 2007-04-18 2014-07-23 基石医药公司 含有硫辛酸衍生物的药物制剂
WO2008137470A1 (fr) 2007-05-01 2008-11-13 Pgr-Solutions Polyamines lipophiles à chaînes multiples
US20090163705A1 (en) 2007-05-21 2009-06-25 Alnylam Pharmaceuticals, Inc. Cationic lipids
WO2009030254A1 (fr) 2007-09-04 2009-03-12 Curevac Gmbh Complexes d'arn et de peptides cationiques pour transfection et immunostimulation
WO2009036280A1 (fr) 2007-09-12 2009-03-19 Copernicus Therapeutics, Inc. Expression de transgène in vivo à long terme
JP5697450B2 (ja) 2007-10-02 2015-04-08 マリーナ バイオテック，インコーポレイテッド 核酸の送達のためのリポペプチド
WO2009046739A1 (fr) 2007-10-09 2009-04-16 Curevac Gmbh Composition pour traiter le cancer de la prostate (pca)
EP2500427B1 (fr) 2007-11-22 2014-07-30 Japan Science and Technology Agency Système de régulation de la traduction dans un modèle cellulaire ou cellulaire artificiel utilisant de l'ARN à faible poids moléculaire
EP3705125B1 (fr) 2007-12-04 2023-07-05 Alnylam Pharmaceuticals, Inc. Conjugués glucidiques utilisés en tant qu'agents d'administration pour des oligonucléotides
CA2710713C (fr) 2007-12-27 2017-09-19 Protiva Biotherapeutics, Inc. Silencage de l'expression de la polo-like kinase a l'aide d'un arn interferent
US20110038941A1 (en) 2007-12-27 2011-02-17 The Ohio State University Research Foundation Lipid Nanoparticle Compositions and Methods of Making and Using the Same
CA3252166A1 (en) 2008-01-02 2025-11-29 Arbutus Biopharma Corp Screening method for selected amino lipid-containing compositions
AU2009234266B2 (en) 2008-04-11 2015-08-06 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
US8058069B2 (en) 2008-04-15 2011-11-15 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
US20090263407A1 (en) 2008-04-16 2009-10-22 Abbott Laboratories Cationic Lipids and Uses Thereof
WO2009127230A1 (fr) 2008-04-16 2009-10-22 Curevac Gmbh Arn(m) modifié pour supprimer ou éviter une réponse immunostimulante et composition immunosuppressive
CA2726052A1 (fr) 2008-06-04 2009-12-10 The Board Of Regents Of The University Of Texas System Modulation de l'expression genique par ciblage d'un petit arn endogene de promoteurs de genes
JP5024216B2 (ja) 2008-07-23 2012-09-12 トヨタ自動車株式会社 内燃機関の点火時期制御装置及び点火時期制御方法
US20100035249A1 (en) 2008-08-05 2010-02-11 Kabushiki Kaisha Dnaform Rna sequencing and analysis using solid support
CN102203605B (zh) 2008-08-27 2014-07-23 生命技术公司 处理生物样品的设备和方法
WO2010037408A1 (fr) 2008-09-30 2010-04-08 Curevac Gmbh Composition comprenant un arnm complexé et un arnm nu pour déclencher ou augmenter une réponse immunostimulante chez un mammifère et utilisations de ladite composition
WO2010042877A1 (fr) 2008-10-09 2010-04-15 Tekmira Pharmaceuticals Corporation Lipides aminés améliorés et procédés d'administration d'acides nucléiques
EP2349210B1 (fr) 2008-10-16 2015-03-18 Marina Biotech, Inc. Procédés et compositions pour une administration liposomale et efficace de produits thérapeutiques de silençage de gène
US9080211B2 (en) 2008-10-24 2015-07-14 Epicentre Technologies Corporation Transposon end compositions and methods for modifying nucleic acids
US20120009222A1 (en) 2008-10-27 2012-01-12 Massachusetts Institute Of Technology Modulation of the immune response
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
CN105152939A (zh) 2008-11-10 2015-12-16 阿尔尼拉姆医药品有限公司 用于递送治疗剂的脂质和组合物
WO2010056403A1 (fr) 2008-11-17 2010-05-20 Enzon Pharmaceuticals, Inc. Lipides cationiques ramifiés pour système d'administration d'acides nucléiques
WO2010083615A1 (fr) 2009-01-26 2010-07-29 Protiva Biotherapeutics, Inc. Compositions et procédés d'inactivation de l'expression de l'apolipoprotéine c-iii
US20100222489A1 (en) 2009-02-27 2010-09-02 Jiang Dayue D Copolymer composition, membrane article, and methods thereof
CN102421900B (zh) 2009-03-12 2015-07-22 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因表达的脂质配制的组合物以及方法
CN102334237B (zh) 2009-04-02 2015-05-13 西蒙公司 电信接线板
ES2927225T3 (es) 2009-04-17 2022-11-03 Univ Oxford Innovation Ltd Composición para entrega de material genético
WO2010124004A2 (fr) 2009-04-22 2010-10-28 Emory University Thérapie par nanosupport pour traiter des tumeurs invasives
KR20210031549A (ko) 2009-05-05 2021-03-19 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
KR20230098713A (ko) 2009-06-10 2023-07-04 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2010147992A1 (fr) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Procédés pour augmenter l'efficacité d'arnsi dans une formulation lipidique
EP2442792A4 (fr) 2009-06-15 2015-12-23 Alnylam Pharmaceuticals Inc Arn double brin en formulation lipidique ciblant le gène pcsk9
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
EP2449106B1 (fr) 2009-07-01 2015-04-08 Protiva Biotherapeutics Inc. Compositions et procédés permettant le silençage de l'apolipoprotéine b
WO2011011447A1 (fr) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions et méthodes pour rendre silencieuse l’expression du gène du virus ebola
US9040701B2 (en) 2009-07-30 2015-05-26 Laboratorios Salvat, S.A. Apaf-1 inhibitor compounds
DE102009043342A1 (de) 2009-09-29 2011-03-31 Bayer Technology Services Gmbh Stoffe für selbstorganisierte Träger zur kontrollierten Freisetzung eines Wirkstoffs
EP3403647A1 (fr) 2009-12-01 2018-11-21 Translate Bio, Inc. Administration d'arnm pour l'augmentation des protéines et des enzymes dans des maladies génétiques humaines
EP3287525B1 (fr) 2009-12-07 2019-10-23 The Trustees of The University of Pennsylvania Préparations d'arn comprenant de l'arn modifié purifié de reprogrammation de cellules
WO2011069529A1 (fr) 2009-12-09 2011-06-16 Curevac Gmbh Solution contenant du mannose pour la lyophilisation, la transfection et/ou l'injection d'acides nucléiques
CA2784568A1 (fr) 2009-12-18 2011-06-23 Martin A. Maier Particules de lipide destinees a la distribution d'acides nucleiques
EP2338520A1 (fr) 2009-12-21 2011-06-29 Ludwig Maximilians Universität Conjugué doté d'un liguant de recherche de cible et son utilisation
EP3721943A1 (fr) 2009-12-23 2020-10-14 Novartis AG Lipides, compositions de lipides et leurs procédés d'utilisation
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
NZ605079A (en) 2010-06-03 2015-08-28 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
CN101863544B (zh) 2010-06-29 2011-09-28 湖南科技大学 一种氰尿酸基重金属螯合絮凝剂及其制备方法
WO2012000104A1 (fr) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Systèmes non liposomaux pour une administration d'acide nucléique
US20130323269A1 (en) 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
CA2807552A1 (fr) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Acides nucleiques modifies et leurs procedes d'utilisation
WO2012019630A1 (fr) 2010-08-13 2012-02-16 Curevac Gmbh Acide nucléique comprenant ou codant pour une tige-boucle d'histone et une séquence poly(a) ou un signal de polyadénylation pour augmenter l'expression d'une protéine codée
WO2012027675A2 (fr) 2010-08-26 2012-03-01 Massachusetts Institute Of Technology Poly(bêta-amino-alcools), leur préparation et utilisations de ceux-ci
EP3431485B2 (fr) 2010-10-01 2024-09-04 ModernaTX, Inc. Acides nucléiques techniques et leurs procédés d'utilisation
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
WO2012113413A1 (fr) 2011-02-21 2012-08-30 Curevac Gmbh Composition de vaccin comprenant des acides nucléiques immunostimulateurs complexés et des antigènes emballés avec des conjugués de polyéthylèneglycol/peptide à liaison disulfure
US9238716B2 (en) 2011-03-28 2016-01-19 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
WO2012135805A2 (fr) 2011-03-31 2012-10-04 modeRNA Therapeutics Administration et formulation d'acides nucléiques génétiquement modifiés
WO2012133737A1 (fr) 2011-03-31 2012-10-04 公益財団法人地球環境産業技術研究機構 Composé amine réticulable, membrane polymère utilisant un composé amine réticulable, et procédé de production de membrane polymère
US11767534B2 (en) 2011-05-04 2023-09-26 The Broad Institute, Inc. Multiplexed genetic reporter assays and compositions
EP2710136A4 (fr) 2011-05-17 2015-01-21 Moderna Therapeutics Inc Acides nucléiques manipulés et leurs procédés d'utilisation pour des vertébrés non humains
EP2532649B1 (fr) 2011-06-07 2015-04-08 Incella GmbH Lipides aminés, leur synthèse et utilisations associées
CA3198966A1 (fr) 2011-06-08 2012-12-13 Translate Bio, Inc. Lipides clivables
US9862926B2 (en) 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
WO2013039861A2 (fr) 2011-09-12 2013-03-21 modeRNA Therapeutics Acides nucléiques modifiés et leurs procédés d'utilisation
WO2013039857A1 (fr) 2011-09-12 2013-03-21 modeRNA Therapeutics Acides nucléiques modifiés et leurs procédés d'utilisation
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2763701B1 (fr) 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Nucléosides, nucléotides et acides nucléiques modifiés, et leurs utilisations
RU2014117690A (ru) 2011-10-05 2015-11-10 Протива Байотерапьютикс Инк. Композиции и способы для сайленсинга альдегид-дегидрогеназы
PE20181541A1 (es) 2011-10-27 2018-09-26 Massachusetts Inst Technology Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco
US20140343129A1 (en) 2011-12-14 2014-11-20 Moderna Therapeutics, Inc. Modified nucleic acids, and acute care uses thereof
EP2791364A4 (fr) 2011-12-14 2015-11-11 Moderna Therapeutics Inc Procédés de réponse à une menace biologique
KR20140102759A (ko) 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물
JP2015510495A (ja) 2011-12-21 2015-04-09 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 器官または器官移植片の生存可能性または寿命を延長する方法
WO2013101690A1 (fr) 2011-12-29 2013-07-04 modeRNA Therapeutics Arnm modifies codant pour des polypeptides pénétrant dans les cellules
EP3677678B1 (fr) 2011-12-30 2024-01-31 Cellscript, Llc Fabrication et utilisation d'un arnss synthétisé in vitro à introduire dans des cellules de mammifères pour induire un effet biologique ou biochimique
WO2013120497A1 (fr) 2012-02-15 2013-08-22 Curevac Gmbh Acide nucléique comprenant ou codant pour une tige-boucle d'histone et une séquence poly(a) ou un signal de polyadénylation pour l'augmentation de l'expression d'une protéine thérapeutique codée
WO2013126803A1 (fr) 2012-02-24 2013-08-29 Protiva Biotherapeutics Inc. Lipides cationiques trialkylés et leurs procédés d'utilisation
EP2830596B1 (fr) 2012-03-29 2020-12-30 Translate Bio, Inc. Nanoparticules neutres dérivées de lipides
ES2762873T3 (es) 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
DE18200782T1 (de) 2012-04-02 2021-10-21 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen
US20150050354A1 (en) 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
US20140275229A1 (en) 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EP2859102A4 (fr) 2012-06-08 2016-05-11 Shire Human Genetic Therapies Polynucléotides résistant à la nucléase et leurs utilisations
WO2014028487A1 (fr) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Lipidoïdes contenant des amines et leurs utilisations
WO2014093924A1 (fr) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Molécules d'acide nucléique modifiées et leurs utilisations
CA2892529C (fr) 2012-11-26 2023-04-25 Moderna Therapeutics, Inc. Arn modifie a son extremite terminale
US20150366997A1 (en) 2012-12-07 2015-12-24 Shire Human Genetics Therapies, Inc. COMPOSITIONS AND METHODS FOR mRNA DELIVERY
WO2014093574A1 (fr) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Polynucléotides modifiés pour modifier le phénotype cellulaire
HK1217215A1 (zh) 2013-01-17 2016-12-30 Modernatx, Inc. 用於改变细胞表型的信号传感器多核苷酸
US20160022774A1 (en) 2013-03-12 2016-01-28 Moderna Therapeutics, Inc. Diagnosis and treatment of fibrosis
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
WO2014152659A1 (fr) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Évaluation quantitative pour l'efficacité de coiffage de l'arn messager
ES2981185T3 (es) 2013-03-14 2024-10-07 Translate Bio Inc Métodos para la purificación de ARN mensajero
WO2014152211A1 (fr) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation et administration de compositions de nucléosides, de nucléotides, et d'acides nucléiques modifiés
US10266559B2 (en) 2013-03-14 2019-04-23 Translate Bio, Inc. Ribonucleic acids with 4′-thio-modified nucleotides and related methods
EP3431592A1 (fr) 2013-03-14 2019-01-23 Translate Bio, Inc. Compositions thérapeutiques d'arnm et utilisation dans le traitement de maladies et de troubles
CA2904151C (fr) 2013-03-14 2023-09-12 Shire Human Genetic Therapies, Inc. Composition a base d'arnm du gene cftr et procedes et utilisations associes
MX2015011945A (es) 2013-03-14 2015-12-01 Shire Human Genetic Therapies Evaluacion cuantitativa para la eficacia de los casquetes de arn mensajero.
LT2970456T (lt) 2013-03-14 2021-08-10 Translate Bio, Inc. Būdai ir kompozicijos, skirti mrnr koduojamų antikūnų pristatymui
WO2014144767A1 (fr) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Purification d'arnm par échange d'ions
PL4332576T3 (pl) 2013-03-15 2025-09-08 Translate Bio, Inc. Synergistyczne wzmocnienie dostarczania kwasów nukleinowych poprzez formulacje zmieszane
EP2971033B8 (fr) 2013-03-15 2019-07-10 ModernaTX, Inc. Procédés de fabrication pour la production de transcrits d'arn
US20160017313A1 (en) 2013-03-15 2016-01-21 Moderna Therapeutics, Inc. Analysis of mrna heterogeneity and stability
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014144039A1 (fr) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Caractérisation de molécules d'arnm
WO2014179562A1 (fr) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology Dérivés de 1,3,5-triazinane-2,4,6-trione et leurs utilisations
US9895443B2 (en) 2013-06-26 2018-02-20 Massachusetts Institute Of Technology Multi-tailed lipids and uses thereof
DK3019619T3 (da) 2013-07-11 2021-10-11 Modernatx Inc Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder
JP6620093B2 (ja) 2013-07-23 2019-12-11 アービュートゥス バイオファーマ コーポレイションＡｒｂｕｔｕｓ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐｏｒａｔｉｏｎ メッセンジャーｒｎａを送達するための組成物及び方法
EP3041934A1 (fr) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Polynucléotides chimériques
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
US20160264614A1 (en) 2013-10-02 2016-09-15 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
EP3052511A4 (fr) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Molécules de polynucléotides et leurs utilisations
WO2015058069A1 (fr) 2013-10-18 2015-04-23 Moderna Therapeutics, Inc. Compositions et procédés pour tolériser des systèmes cellulaires
WO2015061491A1 (fr) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Thérapie à l'arnm pour la phénylcétonurie
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
US20150110857A1 (en) 2013-10-22 2015-04-23 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
EP3076994A4 (fr) 2013-12-06 2017-06-07 Modernatx, Inc. Vaccins adaptatifs ciblés
US20150167017A1 (en) 2013-12-13 2015-06-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
AU2014375402B2 (en) 2013-12-30 2020-10-01 CureVac SE Artificial nucleic acid molecules
US20170002060A1 (en) 2014-01-08 2017-01-05 Moderna Therapeutics, Inc. Polynucleotides for the in vivo production of antibodies
ES2754239T3 (es) 2014-03-12 2020-04-16 Curevac Ag Combinación de vacunación y agonistas de OX40
DK4023249T3 (da) 2014-04-23 2025-01-13 Modernatx Inc Nukleinsyrevacciner
KR20170075742A (ko) 2014-10-02 2017-07-03 프로티바 바이오쎄라퓨틱스, 인코포레이티드 B형 간염 바이러스 유전자 발현을 제거하는 조성물 및 방법
WO2016071857A1 (fr) 2014-11-07 2016-05-12 Protiva Biotherapeutics, Inc. Compositions et méthodes pour le silençage de l'expression du virus ebola
EP3041948B1 (fr) 2014-11-10 2019-01-09 Modernatx, Inc. Molécules d'acide nucléique de remplacement contenant une quantité réduite d'uracile et utilisations associées
WO2016077123A1 (fr) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Optimisation d'acides nucléiques à plusieurs paramètres
EP3247363A4 (fr) 2015-01-21 2018-10-03 Moderna Therapeutics, Inc. Compositions de nanoparticules lipidiques
WO2016118725A1 (fr) 2015-01-23 2016-07-28 Moderna Therapeutics, Inc. Compositions de nanoparticules lipidiques
US20180245077A1 (en) 2015-03-20 2018-08-30 Protiva Biotherapeutics, Inc. Compositions and methods for treating hypertriglyceridemia
WO2016164762A1 (fr) 2015-04-08 2016-10-13 Moderna Therapeutics, Inc. Polynucléotides codant pour des mutants, au niveau des domaines egf-a et intracellulaire, du récepteur des lipoprotéines basse densité et et leurs procédés d'utilisation
WO2016183366A2 (fr) 2015-05-12 2016-11-17 Protiva Biotherapeutics, Inc. Compositions et méthodes permettant l'extinction de l'expression de l'arn du virus de l'hépatite d
WO2016197132A1 (fr) 2015-06-04 2016-12-08 Protiva Biotherapeutics Inc. Traitement d'une infection à virus de l'hépatite b à de l'aide de crispr
WO2016197133A1 (fr) 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Administration d'agents thérapeutiques crispr avec des nanoparticules de lipide
WO2016201377A1 (fr) 2015-06-10 2016-12-15 Moderna Therapeutics, Inc. Vaccins adaptatifs ciblés
WO2017019891A2 (fr) 2015-07-29 2017-02-02 Protiva Biotherapeutics, Inc. Compositions et méthodes de silençage de l'expression du gène du virus de l'hépatite b
US12109274B2 (en) 2015-09-17 2024-10-08 Modernatx, Inc. Polynucleotides containing a stabilizing tail region
US11434486B2 (en) 2015-09-17 2022-09-06 Modernatx, Inc. Polynucleotides containing a morpholino linker
WO2017049074A1 (fr) 2015-09-18 2017-03-23 Moderna Therapeutics, Inc. Formulations de polynucléotides à utiliser dans le traitement de néphropathies
BR112020021037A2 (pt) 2018-04-25 2021-01-19 Ethris Gmbh Composição, composição sólida, processo para a preparação de uma composição, método para preservar uma formulação de nano ou micropartícula de um agente terapeuticamente ativo, uso de um composto selecionado de alcanos c3-c5 substituídos com um ou dois grupos hidróxi, e, dispositivo

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999014346A2 (fr) * 1997-09-19 1999-03-25 Sequitur, Inc. THERAPIES GENIQUES A BASE D'ARNm SENS
WO2007024708A2 (fr) * 2005-08-23 2007-03-01 The Trustees Of The University Of Pennsylvania Arn contenant des nucleosides modifies, et procedes d'utilisation associes
WO2008045548A2 (fr) * 2006-10-12 2008-04-17 Copernicus Therapeutics Inc. Cftr à optimisation par codon
WO2011012316A2 (fr) * 2009-07-31 2011-02-03 Ludwig-Maximilians-Universität Arn ayant une combinaison de nucléotides non modifiés et modifiés pour l'expression protéique
WO2012170930A1 (fr) * 2011-06-08 2012-12-13 Shire Human Genetic Therapies, Inc Compositions de nanoparticules lipides et procédés pour le transfert d'arnm
WO2013182683A1 (fr) * 2012-06-08 2013-12-12 Ethris Gmbh Administration pulmonaire d'un arn messager
WO2013185069A1 (fr) * 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Administration pulmonaire d'arnm à des cellules cibles autres que pulmonaires

Also Published As

Publication number Publication date
KR102311614B1 (ko) 2021-10-08
IL282453A (en) 2021-06-30
US20160106772A1 (en) 2016-04-21
US11510937B2 (en) 2022-11-29
SG11201507391VA (en) 2015-10-29
JP6316930B2 (ja) 2018-04-25
JP2018100307A (ja) 2018-06-28
CN105142676B (zh) 2022-06-28
JP2023054353A (ja) 2023-04-13
EA037922B1 (ru) 2021-06-07
ES2689523T3 (es) 2018-11-14
JP2016517437A (ja) 2016-06-16
IL240982A0 (en) 2015-11-30
HK1220137A1 (en) 2017-04-28
KR20210122917A (ko) 2021-10-12
WO2014153052A9 (fr) 2015-08-06
IL290953B1 (en) 2023-09-01
MX393573B (es) 2025-03-21
SI2968586T1 (sl) 2018-12-31
CY1120790T1 (el) 2019-12-11
CN105142676A (zh) 2015-12-09
CL2015002675A1 (es) 2016-06-10
EP3446712A8 (fr) 2019-04-10
IL290953A (en) 2022-04-01
AU2021215095A1 (en) 2021-08-26
AU2021215095B2 (en) 2024-09-19
AU2014236305A1 (en) 2015-09-24
HUE042640T2 (hu) 2019-07-29
MX365409B (es) 2019-05-31
US9181321B2 (en) 2015-11-10
IL262984B (en) 2021-05-31
AU2019202582B2 (en) 2021-05-27
NZ711657A (en) 2021-01-29
BR112015022868B1 (pt) 2023-05-16
CA2904151A1 (fr) 2014-09-25
JP2020169218A (ja) 2020-10-15
US10420791B2 (en) 2019-09-24
IL305374A (en) 2023-10-01
SG10201710253QA (en) 2018-01-30
CN115154620A (zh) 2022-10-11
RS57739B1 (sr) 2018-12-31
SMT201800546T1 (it) 2019-01-11
US9713626B2 (en) 2017-07-25
JP7236525B2 (ja) 2023-03-09
PL2968586T3 (pl) 2019-01-31
KR102248744B1 (ko) 2021-05-06
EA202190410A1 (ru) 2022-03-31
PE20151773A1 (es) 2015-12-20
AU2019202582A1 (en) 2019-07-25
US20230302039A1 (en) 2023-09-28
EA201591477A1 (ru) 2016-03-31
NZ751462A (en) 2021-07-30
HK1218068A1 (zh) 2017-02-03
JP6738366B2 (ja) 2020-08-12
EP3446712A1 (fr) 2019-02-27
IL262984A (en) 2018-12-31
US20150038556A1 (en) 2015-02-05
IL282453B (en) 2022-03-01
JP2022022419A (ja) 2022-02-03
IL290953B2 (en) 2024-01-01
WO2014153052A2 (fr) 2014-09-25
KR20210049986A (ko) 2021-05-06
US20200046752A1 (en) 2020-02-13
KR20160010398A (ko) 2016-01-27
CA2904151C (fr) 2023-09-12
UA117008C2 (uk) 2018-06-11
MX2015012333A (es) 2016-05-31
DK2968586T3 (en) 2018-10-08
AU2014236305B2 (en) 2019-01-17
BR112015022868A2 (pt) 2017-11-07
LT2968586T (lt) 2018-11-26
EP2968586A2 (fr) 2016-01-20
HRP20181580T1 (hr) 2019-02-22
US20180015116A1 (en) 2018-01-18
PT2968586T (pt) 2018-11-13
EP2968586B1 (fr) 2018-07-25
IL240982B (en) 2018-11-29

Similar Documents

Publication Publication Date Title
WO2014153052A3 (fr) Composition à base d'arnm du gène cftr et procédés et utilisations associés
EP3458074A4 (fr) Compositions destinées à l'administration d'arnt sous la forme de nanoparticules et procédé d'utilisation associé
ZA201506682B (en) Compositions and methods for the improved production and delivery of rna
WO2012109215A3 (fr) Appareils et procédés de production de microfibres et de nanofibres
WO2013177419A3 (fr) Compositions de nanoparticules lipidiques ainsi que procédés de fabrication et procédés d'utilisation de celles-ci
WO2016029191A3 (fr) Modulateurs de canaux
EP3060611A4 (fr) Composition de revêtement aqueuse et son procédé de fabrication
FI20115758A0 (fi) Uusia viljatuotteita, niiden valmistus ja käyttö, ja niitä sisältäviä kosmeettisia formulaatioita
WO2015044782A3 (fr) Dhe intranasale pour le traitement de maux de tête
EP2672957A1 (fr) Nouvelle composition pour le traitement de la mucoviscidose
EP3325608A4 (fr) Procédés et micro-organismes de production de 1,3-butanediol
EP3049066A4 (fr) Compositions et procédés de fabrication de nanoparticules
EP3043811A4 (fr) Compositions et procédés pour l'introduction de molécules dans des cellules vivantes
IN2014DN04773A (fr)
EP3139949A4 (fr) Procédés et compositions pour le traitement de la fibrose kystique
EP3149035A4 (fr) Compositions thérapeutiques comprenant de petites molécules thérapeutiques et leurs utilisations
SMT202200275T1 (it) Nanoparticelle quali veicoli di rilascio di principi attivi e metodi per la loro produzione
EP3066167A4 (fr) Composition de peinture sans primaire, procédés pour sa fabrication et articles la comprenant
EP3068217A4 (fr) Vecteur de ciblage génique du gène 2 activant la recombinaison, production de porcs miniatures de type scid par ciblage génique à médiation par les talen et utilisation correspondante
EP2833891A4 (fr) Matériaux et méthodes pour le traitement de la fibrose kystique et de l'induction de la sécrétion d'ions
EP2885345B8 (fr) Compositions azéotropiques et de type azéotrope de 2,3,3,4,4,4-hexafluoro-1-butène et de 1,1,1,2,3,3-hexafluoropropane et leurs utilisations
WO2014184658A3 (fr) Bulles stables par absorption de particules par interaction électrostatique
WO2017106683A3 (fr) Molécules d'arn concatémères, compositions, et leurs procédés et utilisations
WO2015171643A3 (fr) Expression de canaux ioniques voltage-dépendants chez les ciliés
HK40101439A (zh) 基因构建体、表达载体、药物组合物及其用途

Legal Events

Date Code Title Description
WWE Wipo information: entry into national phase

Ref document number: 201480015043.5

Country of ref document: CN

121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 14718284

Country of ref document: EP

Kind code of ref document: A2

WWE Wipo information: entry into national phase

Ref document number: 240982

Country of ref document: IL

ENP Entry into the national phase

Ref document number: 20157023977

Country of ref document: KR

Kind code of ref document: A

ENP Entry into the national phase

Ref document number: 2904151

Country of ref document: CA

ENP Entry into the national phase

Ref document number: 2016502924

Country of ref document: JP

Kind code of ref document: A

WWE Wipo information: entry into national phase

Ref document number: 2014718284

Country of ref document: EP

WWE Wipo information: entry into national phase

Ref document number: 201591477

Country of ref document: EA

WWE Wipo information: entry into national phase

Ref document number: A201508754

Country of ref document: UA

WWE Wipo information: entry into national phase

Ref document number: MX/A/2015/012333

Country of ref document: MX

WWE Wipo information: entry into national phase

Ref document number: 001986-2015

Country of ref document: PE

ENP Entry into the national phase

Ref document number: 2014236305

Country of ref document: AU

Date of ref document: 20140314

Kind code of ref document: A

REG Reference to national code

Ref country code: BR

Ref legal event code: B01A

Ref document number: 112015022868

Country of ref document: BR

ENP Entry into the national phase

Ref document number: 112015022868

Country of ref document: BR

Kind code of ref document: A2

Effective date: 20150911

WWW Wipo information: withdrawn in national office

Ref document number: 305374

Country of ref document: IL